RARE DISEASE DAY 2017 – FRIEDREICH ATAXIA:RESEARCHERS AT FRATAGENE THERAPEUTICS AND UNVERSITY OF ROME “TOR VERGATA” HAVE IDENTIFIED A NEW THERAPEUTIC TARGET

Researchers at Fratagene Therapeutics, led by Dr. Alessandra Rufini, in collaboration with researchers at the Department of Biomedicine and Prevention of the University of Rome “Tor Vergata”, coordinated by Prof. Roberto Testi, have identified a new therapeutic target relevant for Friedreich ataxia (FA).

FA is a rare disease, a genetic neurodegenerative affection of children and young adults that progressively leads to disability and that still lacks an approved therapy. The discovery of a new therapeutic target, the enzyme RNF126, paves the way to the development of a new class of drugs able to restore the levels of frataxin, the protein defective in FA, therefore providing hope of cure. The results of this work have just been published in the prestigious journal Cell Reports.
Fratagene Therapeutics is a biotech company created by Prof. Roberto Testi, at the Department of Biomedicine and Prevention of the University of Rome “Tor Vergata”, with the aim of attracting the necessary resources to the development of a cure for FA. Fratagene Therapeutics is part of a long-term research endehavour in innovative drug discovery carried out by Prof. Testi group, funded by Telethon, by the Friedreich Ataxia Research Alliance USA and by two European Research Council grants (an Advanced Grant and a Proof-of-Concept grant).

Benini et a., http://www.cell.com/cell-reports/fulltext/S2211-1247(17)30149-3